Improving outcome measures in late onset Pompe disease: Modified Rasch‐Built Pompe‐Specific Activity scale von Kooten, Harmke A., Horton, Mike C., Wenninger, Stephan, Babačić, Haris, Schoser, Benedikt, Lefeuvre, Claire, Taouagh, Najib, Laforêt, Pascal, Segovia, Sonia, Díaz‐Manera, Jordi, Claeys, Kristl G., Mongini, Tiziana, Musumeci, Olimpia, Toscano, Antonio, Hundsberger, Thomas, Brusse, Esther, Doorn, Pieter A., Ploeg, Ans T., Beek, Nadine A. M. E., Kooten, H. A, Brusse, E., Doorn, P. A, Ploeg, A. T., Beek, N. A. M. E., Wenninger, S., Babačić, H., Schoser, B., Montagnese, F., Gracia Angarita, N., Lefeuvre, C., Taouagh, N., Laforêt, P., Béhin, A., Tard, C., Campana‐Salort, E., Sacconi, S., Solé, G., Spinazzi, M., Bouhour, F., Bouibede, F., Hamroun, D., Hogrel, J. Y., Segovia, S., Díaz‐Manera, J., Claeys, K.G., Mongini, T., Musumeci, O., Toscano, A., Hundsberger, T., Horton, M. C.

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The French Pompe registry. Baseline characteristics of a cohort of 126 patients with adult Pompe disease von Laforêt, P., Laloui, K., Granger, B., Hamroun, D., Taouagh, N., Hogrel, J.-Y., Orlikowski, D., Bouhour, F., Lacour, A., Salort-Campana, E., Penisson-Besnier, I., Sacconi, S., Zagnoli, F., Chapon, F., Eymard, B., Desnuelle, C., Pouget, J.

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Cytokine and chemokine profiling shows rapid activation of the immune system following enzyme replacement therapy in Pompe disease von Masat, E, Laforêt, P, Amelin, D, Veron, P, Perniconi, B, Taouagh, N, Benveniste, O, Mingozzi, F

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The impact of enzyme replacement therapy on the progression of Pompe disease von De Antonio, M, Hamroun, D, Perniconi, B, Taouagh, N, Salort-Campana, E, Sacconi, S, Zagnoli, F, Laforêt, P

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Vaginal birth in a patient with Pompe disease von Perniconi, B, Vauthier-Brouzes, D, Morelot-Panzini, C, Dommergues, M, Nizard, J, Taouagh, N, Servais, L, Laforêt, P

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Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease von Papadopoulos, C, Orlikowski, D, Prigent, H, Perniconi, B, Taouagh, N, Lacour, A, Tard, C, Furby, A, Praline, J, Solé, G, Semplicini, C, Kaminsky, P, Eymard, B, Hamroun, D, Laforêt, P

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O.23A 10 year prospective study on the effects of enzyme replacement therapy in adult Pompe patients von Harlaar, L., Hogrel, J., Perniconi, B., Kruijshaar, M., Rizopoulos, D., Taouagh, N., Canal, A., Brusse, E., van Doorn, P., van der Ploeg, A., Laforêt, P., van der Beek, N.

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P.398Usefulness of R-Pact scale for the follow-up of patients with late-onset Pompe disease: results from the French Pompe registry von Lefeuvre, C., Bouhour, F., Nadaj-Pakleza, A., Sacconi, S., Salort-Campana, E., Sole, G., Tard, C., De Antonio, M., Taouagh, N., Hamroun, D., Laforet, P., French pompe study group

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G.P.1 - Cytokine and chemokine profiling shows rapid activation of the immune system following enzyme replacement therapy in Pompe disease von Masat, E., Laforêt, P., Amelin, D., Veron, P., Perniconi, B., Taouagh, N., Benveniste, O., Mingozzi, F.

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G.P.9 - The impact of enzyme replacement therapy on the progression of Pompe disease von De Antonio, M., Hamroun, D., Perniconi, B., Taouagh, N., Salort-Campana, E., Sacconi, S., Zagnoli, F., Laforêt, P.

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G.P.2 - Vaginal birth in a patient with Pompe disease von Perniconi, B., Vauthier-Brouzes, D., Morelot-Panzini, C., Dommergues, M., Nizard, J., Taouagh, N., Servais, L., Laforêt, P.

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P.67 - Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease von Papadopoulos, C., Orlikowski, D., Prigent, H., Perniconi, B., Taouagh, N., Lacour, A., Tard, C., Furby, A., Praline, J., Solé, G., Semplicini, C., Kaminsky, P., Eymard, B., Hamroun, D., Laforêt, P.

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The French Pompe registry. Baseline characteristics of a cohort of 126 patients with adult Pompe disease: Affections musculaires von LAFORET, P, LALOUI, K, PENISSON-BESNIER, I, SACCONI, S, ZAGNOLI, F, CHAPON, F, EYMARD, B, DESNUELLE, C, POUGET, J, GRANGER, B, HAMROUN, D, TAOUAGH, N, HOGREL, J.-Y, ORLIKOWSKI, D, BOUHOUR, F, LACOUR, A, SALORT-CAMPANA, E

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Improving outcome measures in late onset Pompe disease: Modified Rasch-Built Pompe-Specific Activity scale von van Kooten, H.A, Horton, M.C, Wenninger, S, Babačić, H, Schoser, B, Lefeuvre, C, Taouagh, N, Laforêt, P, Segovia, S, Díaz‐Manera, J, Claeys, K.G, Mongini, T, Musumeci, O, Toscano, A, Hundsberger, T, Brusse, E, van Doorn, P.A, van der Ploeg, A.T, van der Beek, N.A.M.E, European Pompe Consortium study group on outcome measures

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Late-onset Pompe disease in France: molecular features and epidemiology from a nationwide study von Semplicini, Claudio, Letard, Pascaline, De Antonio, Marie, Taouagh, Nadjib, Perniconi, Barbara, Bouhour, Françoise, Echaniz-Laguna, Andoni, Orlikowski, David, Sacconi, Sabrina, Salort-Campana, Emmanuelle, Solé, Guilhem, Zagnoli, Fabien, Hamroun, Dalil, Froissart, Roseline, Caillaud, Catherine, Laforêt, Pascal

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Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients von Masat, Elisa, Laforêt, Pascal, De Antonio, Marie, Corre, Guillaume, Perniconi, Barbara, Taouagh, Nadjib, Mariampillai, Kuberaka, Amelin, Damien, Mauhin, Wladimir, Hogrel, Jean-Yves, Caillaud, Catherine, Ronzitti, Giuseppe, Puzzo, Francesco, Kuranda, Klaudia, Colella, Pasqualina, Mallone, Roberto, Benveniste, Olivier, Mingozzi, Federico, Bassez, G., Bedat-Millet, A. L., Behin, A., Eymard, B., Leonard-Louis, S., Stojkovic, T., Canal, A., Decostre, V., Bouhour, F., Boyer, F., Castaing, Y., Chapon, F., Cintas, P., Durieu, I., Echaniz-Laguna, A., Feasson, L., Furby, A., Hamroun, D., Ferrer, X., Solé, G., Froissart, R., Piraud, M., Germain, D., Benistan, K., Guffon-Fouilhoux, N., Journel, H., Labauge, P., Lacour, A., Levy, A., Magot, A., Péréon, Y., Minot-Myhié, M. -C., Nadaj-Pakleza, A., Nathier, C., Orlikowski, D., Pellegrini, N., Petiot, P., Praline, J., Lofaso, F., Prigent, H., Dutry, A., Renard, D., Sacconi, S., Desnuelle, C., Salort-Campana, E., Pouget, J., Tiffreau, V., Vincent, D., Zagnoli, F.

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Registre français de la maladie de Pompe : une cohorte de 210 patients adultes von Lefeuvre, Claire, Clément, Guemy, Azzedine, Arrassi, Nadjib, Taouagh, Marie, De Antonio, Nicolas, Guillaume, Laforet, Pascal

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